Sharing and the Provision of "Cost-Ineffective" Life-Extending Services to Less Severely Ill Patients.

BACKGROUND: Cost-utility analysis prioritizes services using cost, life-years, and the health state utility of the life-years. Nevertheless, a significant body of evidence suggests that the public would prefer more variables to be considered in decision making and at least some sharing of the budget with services for severe conditions that are not cost-effective because of their high cost. OBJECTIVES: To examine whether this preference for sharing persists for less severe conditions when both cost effectiveness and illness severity would indicate that resources should be allocated to other services. METHODS: Survey respondents were asked to divide a budget between two patients facing life-threatening illnesses. The severity of the illnesses differed and the price of treatment was varied. RESULTS: Sharing occurred in all scenarios including scenarios in which the illness was less severe and services were not cost-effective. Results are consistent with behavior commonly observed in other contexts. CONCLUSIONS: Results suggest that sharing per se is important and that the public would support some funding of cost-ineffective services for less severe health problems.

Uncertainty and the Undervaluation of Services for Severe Health States in Cost-Utility Analyses.

OBJECTIVES: To test the hypothesis that the "severity effect"-the preference for more than utility-maximizing expenditure on severe health states-may be the result of, or exacerbated by, the uncertainty associated with the chance of contracting the illness that causes the severe health state. METHODS: Survey respondents were asked to imagine that they will contract one of two illnesses and asked to allocate a budget between two insurance policies, each of which provides services for the treatment of one of the illnesses. A person's final health state varied with the amount of insurance purchased for the illness that occurred and therefore with the level of treatment. The relative cost of the two policies was altered and the selected levels of insurance compared with the levels that would be provided by a health authority that sought to maximize total utility or quality-adjusted life-years from its own budget. RESULTS: Respondents selected more than utility-maximizing insurance for protection against severe health states. A number of psychological factors that affect measurement under uncertainty do not affect utility as currently measured. This difference may explain the present results and also explain the "severity paradox" that personal preferences as presently measured imply less expenditure on severe health states than do "social preferences" for the treatment of strangers. CONCLUSIONS: Uncertainty alters preferences. Incorporating these preferences in decision making would result in greater spending on severe health states.

Variation in the apparent importance of health-related problems with the instrument used to measure patient welfare.

AIMS: This paper investigates the distributional implications for eight population groups of using six different instruments to measure wellbeing and to prioritise access to health services. Specifically, it examines the importance of different physical and psycho-social problems for the scores obtained using each instrument and whether scores differ because of differences in the concept measured by the instrument or because of the instrument's construction. METHODS: Patients with seven chronic conditions and a sample of the 'healthy' public were administered six instruments: two utility instruments; two self-rating scales; a subjective wellbeing instrument and the ICECAP measure of capability. Scores were regressed upon the subscales of the SF-36 and the AQoL-8D. Each instrument's 'problem mix' was measured by the numerical importance of the subscales for the instrument's score and compared with the problem mix of patients constructed from all of the instruments. RESULTS: The apparent importance of different problems varied significantly with the instrument used to assess welfare but not with the chronic conditions. The correspondence between an instrument's problem mix and the patients' problem mix was highly variable. CONCLUSION: Different instruments give prominence to different physical and psycho-social problems and consequently favour different groups of patients. Budgetary decisions which appear to be based on efficiency criteria such as the cost per quality-adjusted life year (QALY) conceal distributive effects attributable to the instrument used in the analysis. The effects are additional to the ethical questions considered in making an equity-efficiency trade-off as they arise from the measurement of efficiency.

Assessing the validity of the ICECAP-A capability measure for adults with depression.

BACKGROUND: Effectiveness and cost-effectiveness are increasingly important considerations in determining which mental health services are funded. Questions have been raised concerning the validity of generic health status instruments used in economic evaluation for assessing mental health problems such as depression; measuring capability wellbeing offers a possible alternative. The aim of this study is to assess the validity of the ICECAP-A capability instrument for individuals with depression. METHODS: Hypotheses were developed using concept mapping. Validity tests and multivariable regression analysis were applied to data from a cross-sectional dataset to assess the performance of ICECAP-A in individuals who reported having a primary condition of depression. The ICECAP-A was collected alongside instruments used to measure: 1. depression using the depression scale of the Depression, Anxiety and Stress Scale (DASS-D of DASS-21); 2. mental health using the Kessler Psychological Distress Scale (K10); 3. generic health status using a common measure collected for use in economic evaluations, the five level version of EQ-5D (EQ-5D-5L). RESULTS: Hypothesised associations between the ICECAP-A (items and index scores) and depression constructs were fully supported in statistical tests. In the multivariable analysis, instruments designed specifically to measure depression and mental health explained a greater proportion of the variation in ICECAP-A than the EQ-5D-5L. CONCLUSION: The ICECAP-A instrument appears to be suitable for assessing outcome in adults with depression for resource allocation purposes. Further research is required on its responsiveness and use in economic evaluation. Using a capability perspective when assessing cost-effectiveness could potentially re-orientate resource provision across physical and mental health care services.

Deriving population norms for the AQoL-6D and AQoL-8D multi-attribute utility instruments from web-based data.

OBJECTIVES: (i) to demonstrate a method which ameliorates the problem of self-selection in the estimation of population norms from web-based data and (ii) to use the method to calculate population norms for two multi-attribute utility (MAU) instruments, the AQoL-6D and AQoL-8D, and population norms for the sub-scales from which they are constructed. METHODS: A web-based survey administered the AQoL-8D MAU instrument (which subsumes the AQoL-6D questionnaire), to members of the public along with the AQoL-4D which has extant population norms. Age, gender and the AQoL-4D were used as post-stratification auxiliary variables to construct weights to ameliorate the potential effects of self-selection associated with web-based surveys. The weights were used to estimate unbiased population norms. Standard errors from the weighted samples were calculated using Jackknife estimation. RESULTS: For both AQoL-6D and AQoL-8D, physical health dimensions decline significantly with age. In contrast, for the majority of the psycho-social dimensions there is a significant U-shaped profile. The net effect is a shallow U-shaped relationship between age and both the AQoL-6D and AQoL-8D utilities. This contrasts with the almost monotonic decline in the utilities derived from the AQoL-4D and SF-6D MAU instruments. CONCLUSIONS: Post-stratification weights were used to ameliorate potential bias in the derivation of norms from web-based data for the AQoL-6D and AQoL-8D. The methods may be used generally to obtain norms when suitable auxiliary variables are available. The inclusion of an enlarged psycho-social component in the two instruments significantly alters the demographic profile.

Estimating Utility Weights for the Vision Related Quality of Life Index.

PURPOSE: The VisQoL instrument was constructed as a vision-specific addition to the AQoL-6D multi-attribute utility instrument. The composite instrument, the AQoL-7D, was assigned utility scores that are the basis for now estimating VisQoL utilities when it is used as a stand-alone instrument. This study aimed to construct mapping functions that allow utility scores to be assigned to the Vision Related Quality of Life (VisQoL) instrument, a stand-alone vision-specific quality-of-life measure. METHODS: A sample of 164 patients completed the AQoL-7D, which includes the VisQoL. Mapping algorithms between VisQoL and AQoL-7D were then derived using two econometric methods, ordinary least squares estimator and generalized linear model (GLM). Two model specifications were considered with either six VisQoL raw item values or VisQoL overall dimension value as the key independent variables. The predictive performance of each method on each model specification was assessed using the mean absolute error (MAE) and intraclass correlation coefficient (ICC). Both internal and external validation tests (using a second, independent sample of 164 patients) were performed. RESULTS: The mapping algorithms derived from the GLM had superior properties to the ordinary least squares-based algorithms in both internal and external validation tests. The ICC values ranged from 0.851 to 0.913, and the MAE ranged from 0.043 to 0.052 for two model specifications, based on two econometric methods. However, predicted utilities tend to over-predict/under-predict the lowest/highest observed utility. CONCLUSIONS: Mapping algorithms predicting AQoL-7D utility based on six VisQoL items or VisQoL dimension value have been developed. The algorithm can be used to estimate quality adjusted life years. This allows the VisQoL to be used in cost utility analyses.

Shared medical appointments for Aboriginal and Torres Strait Islander men.

BACKGROUND: Aboriginal and Torres Strait Islander health is generally the worst of any population group in Australia. Inaccessibility to health services is one possible cause of this. Shared medical appointments (SMAs) appear to be a culturally competent and appropriate way of improving access to, and the quality of, primary healthcare services for Aboriginal and Torres Strait Islander peoples. OBJECTIVE: The objective of this article is to assess the acceptability and appropriateness of SMAs as an adjunct process in primary care for Aboriginal and Torres Strait Islander men. METHODS: As part of a broader study on SMAs, three SMA sessions were delivered at an Aboriginal men's health centre in northern New South Wales. One-day training sessions in SMA facilitation were also provided to two groups of 12-14 Aboriginal health workers (AHWs). Mixed methods were used to assess patient and provider satisfaction, subjective outcomes, and operational procedures in the SMA groups, as well as interest in the SMA process by AHWs. RESULTS: Satisfaction with SMAs among Aboriginal men was unanimously positive, with the numbers in the group increasing over time. Patients most enjoyed the 'yarn up' nature of SMAs with peer support, which reduced the 'scary' and culturally 'unnatural' nature of one-on-one consultations with a general practitioner (GP). AHWs who were trained to a level to conduct SMAs saw this as an effective way of improving cultural competence in, and accessibility of, their various Aboriginal health services. DISCUSSION: The results, though not generalisable, suggest that SMAs may offer a culturally safe and appropriate tool to enhance Aboriginal and Torres Strait Islander peoples' access to primary care.

Why do multi-attribute utility instruments produce different utilities: the relative importance of the descriptive systems, scale and 'micro-utility' effects.

PURPOSE: Health state utilities measured by the major multi-attribute utility instruments differ. Understanding the reasons for this is important for the choice of instrument and for research designed to reconcile these differences. This paper investigates these reasons by explaining pairwise differences between utilities derived from six multi-attribute utility instruments in terms of (1) their implicit measurement scales; (2) the structure of their descriptive systems; and (3) 'micro-utility effects', scale-adjusted differences attributable to their utility formula. METHODS: The EQ-5D-5L, SF-6D, HUI 3, 15D and AQoL-8D were administered to 8,019 individuals. Utilities and unweighted values were calculated using each instrument. Scale effects were determined by the linear relationship between utilities, the effect of the descriptive system by comparison of scale-adjusted values and 'micro-utility effects' by the unexplained difference between utilities and values. RESULTS: Overall, 66 % of the differences between utilities was attributable to the descriptive systems, 30.3 % to scale effects and 3.7 % to micro-utility effects. DISCUSSION: Results imply that the revision of utility algorithms will not reconcile differences between instruments. The dominating importance of the descriptive system highlights the need for researchers to select the instrument most capable of describing the health states relevant for a study. CONCLUSIONS: Reconciliation of inconsistent utilities produced by different instruments must focus primarily upon the content of the descriptive system. Utility weights primarily determine the measurement scale. Other differences, attributable to utility formula, are comparatively unimportant.

An instrument for measuring the social willingness to pay for health state improvement.

This paper describes an instrument for measuring the social value of changes in health status, the Relative Social Willingness to Pay. It is a unique combination of measurement attributes designed to minimise cognitive complexity and provide an additional option for measuring 'social value'. Similar to the person trade-off (PTO), it adopts a social perspective and asks respondents to evaluate programmes on behalf of society. Unlike the PTO, trade-offs between the options use dollars, not numbers of patients. Respondents are not, however, asked for their personal willingness to pay. Rather, the opportunity cost of funds spent on one service is as an offsetting reduction in funds for a second service. The amount spent on each service therefore indicates relative, not absolute, value. However, the two services combine to produce one Quality adjusted life year which allows the calculation of a Quality adjusted life year-like unit of social value on a 0-1 scale. A three-stage survey was used to test the instrument's reliability, validity and sensitivity to the framing of the main question. Results indicate that the Relative Social Willingness to Pay produces values similar to but less than the PTO and time trade-off techniques.

Modelling utility weights for the Assessment of Quality of Life (AQoL)-8D.

PURPOSE: The objective of this paper is to describe the four-stage methodology used to obtain utility scores for the Assessment of Quality of Life (AQoL)-8D, a 35-item 8 dimension multi-attribute utility instrument, which was created to achieve a high degree of sensitivity to psycho-social health. METHODS: Data for the analyses were obtained from a representative group of 347 members of the Australian public and from 323 mental health patients each of whom provided VAS and time trade-off valuations of multiple health states. Data were used initially to create multiplicative scoring algorithms for each of the instrument's 8 dimensions and for the overall instrument. Each of the algorithms was then subject to a second-stage econometric 'correction'. RESULTS: Algorithms were successfully created for each of the AQoL-8D's dimensions, for physical and mental 'super-dimensions' and for the overall AQoL-8D instrument. The final AQoL-8D algorithm has good predictive power with respect to the TTO valuations. CONCLUSIONS: The AQoL-8D is a suitable instrument for researchers conducting cost utility analyses generally but, in particular, for the analysis of services affecting psycho-social health.

Priority setting and patient adaptation to disability and illness: outcomes of a qualitative study.

The study examined the question of who should make decisions for a National Health Scheme about the allocation of health resources when the health states of beneficiaries could change because of adaptation. Eight semi-structured small group discussions were conducted. Following focus group theory, interviews commenced with general questions followed by transition questions and ended with a 'focus' or 'key' question. Participants were presented with several scenarios in which patients adapted to their health states. They were then asked their views about the appropriate role of the public, patients and health professionals in making social judgements of quality of life. After discussion and debate, all groups were asked the key question: 'In light of adaptation, who should evaluate quality of life for the purpose of setting priorities in the allocation of health care?' In all groups participants presented strong arguments for and against decision making by patients, the public and health professionals. However, most groups thought a representative body which included a range of perspectives should make the relevant judgements. This is at odds with the recommendations in most national pharmaceutical guidelines. The main conclusion of the paper is that health economists and other researchers should explore the possibility of adopting a deliberative, consensus-based approach to evaluating health-related quality of life when such judgements are to be used to inform priority setting in a public system.

Preferences for the normative basis of health care priority setting: some evidence from two countries.

The present paper concerns the criteria people would prefer for prioritising health programmes. It differs from most empirical studies as subjects were not asked about their personal preferences for programmes per se. Rather, they were asked about the principles that should guide the choice of programmes. Four different principles were framed as arguments for alternative programmes. The results from population surveys in Australia and Norway suggest that people are least supportive of the principle that decision makers should follow the stated preferences of the public. Rather, respondents expressed more support for decisions based upon health maximisation, equality and urgency.

Measuring the Wellbeing of Cancer Patients with Generic and Disease-Specific Instruments.

Different wellbeing measures have been used among cancer patients. This study aimed to first investigate the sensitivity of health state utility (HSU), capability, and subjective wellbeing (SWB) instruments in cancer. A cancer-specific instrument (QLQ-C30) was included and transferred onto the cancer-specific HSU scores. Furthermore, it examined the relative importance of key life domains explaining overall life satisfaction. Data were drawn from the Multi-instrument Comparison survey. Linear regression was used to explore the extent to which the QLQ-C30 sub-scales explain HSU and SWB. Kernel-based Regularized Least Squares (KRLS), a machine learning method, was used to explore the life domain importance of cancer patients. As expected, the QLQ-C30 sub-scales explained the vast majority of the variance in its derived cancer-specific HSU (R2 = 0.96), followed by generic HSU instruments (R2 of 0.65-0.73) and SWB and capability instruments (R2 of 0.33-0.48). The cancer-specific measure was more closely correlated with generic HSU than SWB measures, owing to the construction of these instruments. In addition to health, life achievements, relationships, the standard of living, and future security all play an important role in explaining the overall life satisfaction of cancer patients.

Valuation of Treatments for Rare Diseases: A Systematic Literature Review of Societal Preference Studies.

INTRODUCTION: We sought to synthesize published empirical studies that elicited and characterized societal valuations of orphan drugs and the attributes that may drive different valuations for orphan drugs versus other treatments. METHODS: We conducted a systematic literature review (SLR) in MEDLINE and EMBASE databases up to November 2, 2020. Search terms covered societal preferences and attributes of orphan drugs (e.g., disease prevalence, severity, burden, unmet needs, and benefits). RESULTS: We identified 38 eligible publications: 33 societal preference studies and 5 reviews discussing societal valuations and attributes of orphan drugs. Most publications suggested that a majority of respondents favored allocating funds to more prevalent diseases. However, trade-off studies and discrete-choice experiments found that survey participants chose to allocate resources to orphan drugs even when the cost per unit of health benefit was greater than for therapies for more prevalent diseases. Overall, 19 of 27 studies assessing severity in treatment valuation revealed that respondents prioritized patients with severe diseases over those with milder ones for equal health benefits. Members of the general public tended to prefer treatments for diseases with no alternative or when existing alternatives had limited efficacy over diseases with clear therapeutic alternatives. There was evidence that individuals preferred sharing resources, so no patient was left without treatment. CONCLUSIONS: Our SLR indicates the general public typically attaches greater value to orphan drugs than to other treatments for common diseases. This is not because of rarity per se, but primarily because of disease severity and lack of therapeutic alternatives typically associated with rare diseases.

Orphan drugs are drugs serving a substantial public health need by treating life-threatening or chronically debilitating medical conditions affecting a small number of people with very high unmet needs. We reviewed 38 published studies looking at drug characteristics that may cause people to value orphan drugs differently versus treatments for common conditions. Most people surveyed in these publications favored health care funds going to more prevalent diseases. However, some people preferred funding orphan drugs even when the cost versus health benefit was higher compared with treatments for more common diseases. The majority of studies that investigated the impact of disease severity on the valuation of treatments found that people prioritized patients with severe disease over those with milder disease, for the same extent of health benefit. People also preferred funding treatments for diseases that have no alternative treatments, or treatments with limited benefits, over treatments for diseases with many treatments or more effective treatments. We also found evidence of a societal preference for shared resources, meaning that no patient would be left without treatment, including those who receive limited benefits from health care resources, even if this does not lead to the maximization of health benefits across society. In conclusion, our literature review indicated that the general public attaches greater value to orphan drugs versus treatments for more common diseases, not because of rarity per se, but largely because the rare diseases treated by orphan drugs are often severe and have no or few treatment options.

Utility-based quality of life of overweight and obese adolescents.

OBJECTIVE: To explore the relationship between overweight/obesity and utility in adolescents. METHODS: Data were collected from 2890 adolescents attending 13 secondary schools in the state of Victoria, Australia. The Assessment of Quality of Life 6-Dimension (AQoL-6D) questionnaire was used to measure individual utility. Adolescent's height and weight were measured and weight status categories assigned according to the World Health Organization adolescent growth standards. Multivariate linear regression analyses were undertaken for the whole population and subpopulations of boys and girls to estimate the mean differences in utility scores between 1) overweight and healthy weight and 2) obese and healthy weight adolescents, while controlling for demographic and socioeconomic status variables. RESULTS: The mean age of adolescents was 14.6 years, 56.2% were boys, 22.2% were overweight, and 9.4% were obese. The mean utility of healthy weight adolescents was 0.860. After adjustments, the overweight and obese groups reported significantly lower mean utility scores (differences: -0.018 and -0.059, respectively, relative to the healthy weight group). This can be interpreted as equivalent to a stated willingness to sacrifice 1.8% and 5.9% of a life in perfect health or 2.3% and 6.8% of a life at healthy weight. A significant utility difference associated with overweight was only experienced by girls (-0.039, P = 0.003). Both sexes experienced significant utility differences associated with obesity, but the magnitude was double for girls (-0.084, P < 0.001) relative to boys (-0.041, P = 0.022). CONCLUSION: Utility is lower among overweight and more so among obese adolescents.

The monetary value of a life year: evidence from a qualitative study of treatment costs.

A small number of studies have provided suggestive evidence that the general public rejects the idea of giving higher priority to low-cost patients, in the context of a limited budget, in order to maximise health benefits. The study reported here used semi-structured group discussions to investigate the normative bases of such views among the Australian public. Discussion groups help participants reflect critically upon their own reasoning processes and go some way towards revealing underlying values rather than unreflective preferences. As a part of the exercise, participants were asked to allocate a hospital budget. After discussion and deliberation only three out of 41 chose to allocate all of the money to the low-cost patients. Reasons were not based on conceptual confusion or lack of insight into the implications of the different strategies, but rather on views about fairness, including the importance of giving all groups a 'chance' of being treated and of not removing 'hope' from high-cost patients. The results suggest that as costs rise people are willing to pay more than the minimum cost of a quality-adjusted life year for equity reasons, indicating that caution must be exercised in estimating a single monetary value for a QALY.

Paying the right price for pharmaceuticals: a case study of why the comparator matters.

This article considers the pricing policy for pharmaceuticals in Australia, which is widely seen as having achieved low drug prices. However, compared to New Zealand, the evidence implies that Australia might have improved its performance significantly if it had proactively sought market best pricing. The Australian record suggests that the information sought by authorities may not be sufficient for optimal pricing and that the economic evaluation of pharmaceuticals may be neither necessary nor sufficient for achieving this goal.

Predicting time trade-off health state valuations of adolescents in four Pacific countries using the Assessment of Quality-of-Life (AQoL-6D) instrument.

OBJECTIVES: Pacific Obesity Prevention in Communities (OPIC) is a community-based intervention project targeting adolescent obesity in Australia, New Zealand, Fiji, and Tonga. The Assessment of Quality of Life Mark 2 (AQoL-6D) instrument was completed by 15,481 adolescents to obtain a description of the quality of life associated with adolescent overweight and obesity, and a corresponding utility score for use in a cost-utility analysis of the interventions. This article describes the recalibration of this utility instrument for adolescents in each country. METHODS: The recalibration was based on country-specific time trade-off (TTO) data for 30 multiattribute health states constructed from the AQoL-6D descriptive system. Senior secondary students, in a classroom setting, responded to 10 health state scenarios each. These TTO interviews were conducted for 24 groups, comprising 279 students in the four countries resulting in 2790 completed TTO scores. The TTO scores were econometrically transformed by regressing the TTO scores upon predicted scores from the AQoL-6D to produce country-specific algorithms. The latter incorporated country-specific "corrections" to the Australian adult utility weights in the original AQoL. RESULTS: This article reports two methodological elements not previously reported. The first is the econometric modification of an extant multi-attribute utility instrument to accommodate cultural and other group-specific differences in preferences. The second is the use of the TTO technique with adolescents in a classroom group setting. Significant differences in utility scores were found between the four countries. CONCLUSION: Statistical results indicate that the AQoL-6D can be validly used in the economic evaluation of both the OPIC interventions and other adolescent programs.

Do quality-adjusted life years take account of lost income? Evidence from an Australian survey.

The procedures used in cost utility analysis for eliciting quality of life weights have generally omitted any instruction concerning the level of consumption in a health state, despite the fact that some health states preclude the possibility of normal employment. This introduces ambiguity into the interpretation of quality of life (QoL) scores, and project ranking is sensitive to the subsequent treatment of consumption in the analysis. This article reports the results of a study that questioned 131 respondents to a time trade-off (TTO) interview about their assumptions concerning consumption and the amount of thought given to consumption. Results indicate that, without prompting, most assumed unchanged consumption, implying little bias in existing studies.

Steering without navigation equipment: the lamentable state of Australian health policy reform.

BACKGROUND: Commentary on health policy reform in Australia often commences with an unstated logical error: Australians' health is good, therefore the Australian Health System is good. This possibly explains the disconnect between the options discussed, the areas needing reform and the generally self-congratulatory tone of the discussion: a good system needs (relatively) minor improvement. RESULTS: This paper comments on some issues of particular concern to Australian health policy makers and some areas needing urgent reform. The two sets of issues do not overlap. It is suggested that there are two fundamental reasons for this. The first is the failure to develop governance structures which promote the identification and resolution of problems according to their importance. The second and related failure is the failure to equip the health services industry with satisfactory navigation equipment - independent research capacity, independent reporting and evaluation - on a scale commensurate with the needs of the country's largest industry. These two failures together deprive the health system - as a system - of the chief driver of progress in every successful industry in the 20th Century. CONCLUSION: Concluding comment is made on the National Health and Hospitals Reform Commission (NHHRC). This continued the tradition of largely evidence free argument and decision making. It failed to identify and properly analyse major system failures, the reasons for them and the form of governance which would maximise the likelihood of future error leaning. The NHHRC itself failed to error learn from past policy failures, a key lesson from which is that a major - and possibly the major - obstacle to reform, is government itself. The Commission virtually ignored the issue of governance. The endorsement of a monopolised system, driven by benevolent managers will miss the major lesson of history which is illustrated by Australia's own failures.